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Immune ThrombocytopeniaOBS'CEREVANCENCT05937828

OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia

An observational study of Immune Thrombocytopenia and Autoimmune Hemolytic Anemia, run by University Hospital, Bordeaux at 9 sites in France.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

From 2004, OBS'CEREVANCE is a national real-world prospective clinical cohort of patients with auto-immune cytopenia of pediatric-onset : Immune thrombocytopenia (ITP), Autoimmune Hemolytic anemia (AIHA), or Evans syndrome (all bi or tri cytopenias). Read more about this study

Who is it for?
Ages Up to 17 years
What is being tested?
data collection
How many people
3,500 participants
Where
9 sitesFrance
Study dates
1 Sep 2010to 31 Dec 2029
How long will I take part?
19 Years
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Study population: Each of the 30 hemato-immunologic pediatric unit in France guaranties the prospective in real time inclusion in the cohort of all the children diagnosed in their unit, before 18 years old, with ITP, AIHA or Evans syndrome diagnosis, whatever the date of diagnosis at the date of inclusion. This observational cohort is prospective from the day of initial diagnosis of the auto-immune cytopenia The pediatricians from this rare disease CEREVANCE national network follow national guidelines for evaluation and treatment (PNDS 2009, 2017)

Who may be able to join

  • Diagnosis of ITP, AIHA, Evans Syndrome
  • Onset before the age of 18

Who cannot join

  • Opposition of legal representative or to data collection

Trial locations and contacts

9 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Nathalie ALADJIDI, MD contact

+33 557820261

nathalie.aladjidi@chu-bordeaux.fr

Aurore Capelli contact

0557820877

aurore.capelli@chu-bordeaux.fr

9 sites in 1 country

France9 sites, 9 recruiting

  • CHU Amiens Picardie Service d' Onco-Immuno-Hématologie Pédiatrique

    Amiens

    Valérie LI THIAO TE, lithiaote.valerie@chu-amiens.fr

    Recruiting

  • CHU d'Angers Unité d'Hémato-Oncologie Pédiatrique

    Angers

    Isabelle PELLIER

    ispellier@chu-angers.fr

    Recruiting

  • BESANCON CHU de Besançon Hôpital Jean MINJOZ Unité d'Hémato-Oncologie Pédiatrique, Pédiatrie 1

    Besançon

    Nathalie CHEIKH

    ncheikh@chu-besancon.fr

    Recruiting

  • CHU de Bordeaux - Unité d'Hématologie et d'Oncologie pédiatrique

    Bordeaux

    Nathalie ALADJIDI, nathalie.aladjidi@chu-bordeaux.fr

    Recruiting

  • CHU BREST Hôpital Morvan Unité d'Onco-Hématologie

    Brest

    LIANA CARAUSU, liana.carausu@chu-brest.fr

    Recruiting

  • CHU de Caen Unité d'Onco-Hématologie

    Caen

    Marianna DEPARIS, deparis-m@chu-caen.fr

    Recruiting

  • CHU de Clermont Ferrand

    Clermont-Ferrand

    Eric DORE, edore@chu-clermontferrand.fr

    Recruiting

  • APHP Hôpital Bicêtre Service de Pédiatrie générale

    Paris

    Corinne GUITTON, corinne.guitton@aphp.fr

    Recruiting

  • CH de Cornouaille Service de Pédiatrie

    Quimper

    Philippe VIC, p.vic@ch-cornouaille.fr

    Recruiting

Other trials in France

About this study

Official title: "French National Cohort of Patients With Pediatric-onset of Autoimmune Cytopenia (OBS'CEREVANCE Cohort)"

From 2004, OBS'CEREVANCE is a national real-world prospective clinical cohort of patients with auto-immune cytopenia of pediatric-onset : Immune thrombocytopenia (ITP), Autoimmune Hemolytic anemia (AIHA), or Evans syndrome (all bi or tri cytopenias). Thanks to the collaboration of the 30 French pediatric hematologic centers, this cohort supports all of the Rare Disease Centre CEREVANCE (Centre de Référence National des Cytopénies Auto-Immunes de l'Enfant) missions for care, education and research. Specifically, this original unbiased database allows to describe the long-term health of adult patients, to identify the heterogenous genetic underlying pathophysiologic contexts, and to study the benefit-risk balance of treatments, including the growing development of targeted therapies.

Read the detailed description

Immune thrombocytopenic purpura (ITP) and autoimmune hemolytic anemia (AHAI) are rare childhood diseases that involve autoimmune destruction of platelets and erythrocytes respectively.

They may be associated with an even rarer entity, Evans syndrome (ES). These three conditions are referred to as autoimmune cytopenias (AIC).

In association with CAI, patients may present with various immunopathological (IM) manifestations such as lymphoproliferation, autoimmune autoimmune/autoinflammatory organ diseases that may be absent at the time of at the time of diagnosis of CAI and develop during follow-up.

Since 2004, the CEREVANCE reference center for childhood autoimmune CEREVANCE has been coordinating a national prospective cohort of patients with pediatric-onset CAI including over 1900 patients (data 05/2023).

Thanks to the collaboration of the 30 French pediatric hematologic centers, this cohort supports all of the Rare Disease Centre CEREVANCE (Centre de Référence National des Cytopénies Auto-Immunes de l'Enfant) missions for care, education and research. Specifically, this original unbiased database allows to describe the long-term health of adult patients, to identify the heterogenous genetic underlying pathophysiologic contexts, and to study the benefit-risk balance of treatments, including the growing development of targeted therapies.

Being tested
data collection
Medical subject headings
Purpura, Thrombocytopenic, Idiopathic, Anemia, Hemolytic, Autoimmune, Evans Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • data collectionOther

    description of the long-term health of adult patients, identification of the heterogenous genetic underlying pathophysiologic contexts, study of the benefit-risk balance of treatments, including the growing development of targeted therapies.

Study groups in NCT05937828
Study groupRoleWhat they receive
Immune thrombocytopenia (ITP)Immune thrombocytopenia (ITP) : defined according to the international working group criteria (Rodeghiero et al., Blood 2009).
Receives: Other: data collection
Autoimmune Hemolytic anemia (AIHA)Autoimmune haemolytic anaemia (AIHA) : Hb < 110 g/L with a positive direct antiglobulin test (DAT) and at least one of the following haemolysis criteria: reticulocyte count > 120 G/L, free bilirubin > 17 mmol/L, or haptoglobin < 10 mg/dL.
Receives: Other: data collection
Evans syndrome (all bi or tri cytopenias)Evans syndrome (ES) : simultaneous (less than 1 month) or sequential association of at least two autoimmune cytopenia among ITP, AIHA and autoimmune neutropenia (AIN).
Receives: Other: data collection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. AIC context

    Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID).

    Measured: Baseline

Secondary outcomes (4)

  1. Treatment lines

    Percentage of patients with each treatment by line of treatments

    Measured: every 6 months after baseline up to 19 years

  2. Adverse drug reactions

    Percentage of patients with adverse drug reaction reported by investigators

    Measured: every 6 months after baseline up to 19 years

  3. Events

    Percentage of patients with other events of interest like cancer, infection, thrombosis, death

    Measured: every 6 months after baseline up to 19 years

  4. AIC context

    Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID).

    Measured: every 6 months after baseline up to 19 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Jul 2023
  2. Study start1 Sep 2010
  3. Primary completion31 Dec 2029 (estimated)
  4. Study completion31 Dec 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
3,500 participants (estimated)
Target duration
19 Years
IDs, dates and publications
ClinicalTrials.gov ID
NCT05937828
Sponsor's protocol ID
CHUBX 2023/08
Acronym
OBS'CEREVANCE
Status last verified
Jul 2023
First submitted
14 Mar 2023
Last updated
10 Jul 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Ducassou S, Leverger G, Fernandes H, Chambost H, Bertrand Y, Armari-Alla C, Nelken B, Monpoux F, Guitton C, Leblanc T, Fisher A, Lejars O, Jeziorski E, Fouissac F, Lutz P, Pasquet M, Pellier I, Piguet C, Vic P, Bayart S, Marie-Cardine A, Michel M, Perel Y, Aladjidi N. Benefits of rituximab as a second-line treatment for autoimmune haemolytic anaemia in children: a prospective French cohort study. Br J Haematol. 2017 Jun;177(5):751-758. doi: 10.1111/bjh.14627. Epub 2017 Apr 26.

    PubMed 28444729

  • Aladjidi N, Jutand MA, Beaubois C, Fernandes H, Jeanpetit J, Coureau G, Gilleron V, Kostrzewa A, Lauroua P, Jeanne M, Thiebaut R, Leblanc T, Leverger G, Perel Y. Reliable assessment of the incidence of childhood autoimmune hemolytic anemia. Pediatr Blood Cancer. 2017 Dec;64(12). doi: 10.1002/pbc.26683. Epub 2017 Jul 27.

    PubMed 28748541

  • Penel Page M, Bertrand Y, Fernandes H, Kherfellah D, Leverger G, Leblanc T, Libbrecht C, Michel G, Jeziorski E, Armari-Alla C, Nelken B, Pellier I, Pasquet M, Perel Y, Aladjidi N. Treatment with cyclosporin in auto-immune cytopenias in children: The experience from the French cohort OBS'CEREVANCE. Am J Hematol. 2018 May 14. doi: 10.1002/ajh.25137. Online ahead of print. No abstract available.

    PubMed 29756344

  • Pincez T, Neven B, Le Pointe HD, Varlet P, Fernandes H, Gareton A, Leverger G, Leblanc T, Chambost H, Michel G, Pasquet M, Millot F, Hermine O, Mathian A, Hully M, Zephir H, Hamidou M, Durand JM, Perel Y, Landman-Parker J, Rieux-Laucat F, Aladjidi N. Neurological Involvement in Childhood Evans Syndrome. J Clin Immunol. 2019 Feb;39(2):171-181. doi: 10.1007/s10875-019-0594-3. Epub 2019 Jan 22.

    PubMed 30671780

  • Hadjadj J, Aladjidi N, Fernandes H, Leverger G, Magerus-Chatinet A, Mazerolles F, Stolzenberg MC, Jacques S, Picard C, Rosain J, Fourrage C, Hanein S, Zarhrate M, Pasquet M, Abou Chahla W, Barlogis V, Bertrand Y, Pellier I, Colomb Bottollier E, Fouyssac F, Blouin P, Thomas C, Cheikh N, Dore E, Pondarre C, Plantaz D, Jeziorski E, Millot F, Garcelon N, Ducassou S, Perel Y, Leblanc T, Neven B, Fischer A, Rieux-Laucat F; members of the French Reference Center for Pediatric Autoimmune Cytopenia (CEREVANCE). Pediatric Evans syndrome is associated with a high frequency of potentially damaging variants in immune genes. Blood. 2019 Jul 4;134(1):9-21. doi: 10.1182/blood-2018-11-887141. Epub 2019 Apr 2.

    PubMed 30940614

  • Margot H, Boursier G, Duflos C, Sanchez E, Amiel J, Andrau JC, Arpin S, Brischoux-Boucher E, Boute O, Burglen L, Caille C, Capri Y, Collignon P, Conrad S, Cormier-Daire V, Delplancq G, Dieterich K, Dollfus H, Fradin M, Faivre L, Fernandes H, Francannet C, Gatinois V, Gerard M, Goldenberg A, Ghoumid J, Grotto S, Guerrot AM, Guichet A, Isidor B, Jacquemont ML, Julia S, Khau Van Kien P, Legendre M, Le Quan Sang KH, Leheup B, Lyonnet S, Magry V, Manouvrier S, Martin D, Morel G, Munnich A, Naudion S, Odent S, Perrin L, Petit F, Philip N, Rio M, Robbe J, Rossi M, Sarrazin E, Toutain A, Van Gils J, Vera G, Verloes A, Weber S, Whalen S, Sanlaville D, Lacombe D, Aladjidi N, Genevieve D. Immunopathological manifestations in Kabuki syndrome: a registry study of 177 individuals. Genet Med. 2020 Jan;22(1):181-188. doi: 10.1038/s41436-019-0623-x. Epub 2019 Jul 31.

    PubMed 31363182

  • Ducassou S, Gourdonneau A, Fernandes H, Leverger G, Pasquet M, Fouyssac F, Bayart S, Bertrand Y, Michel G, Jeziorski E, Thomas C, Abouchallah W, Viard F, Guitton C, Cheikh N, Pellier I, Carausu L, Droz C, Leblanc T, Aladjidi N; Centre de Reference National des Cytopenies Auto-immunes de l'Enfant (CEREVANCE). Second-line treatment trends and long-term outcomes of 392 children with chronic immune thrombocytopenic purpura: the French experience over the past 25 years. Br J Haematol. 2020 Jun;189(5):931-942. doi: 10.1111/bjh.16448. Epub 2020 Mar 4.

    PubMed 32130726

  • Ducassou S, Fernandes H, Savel H, Bertrand Y, Leblanc T, Abou Chahla W, Pasquet M, Leverger G, Barlogis V, Thomas C, Bayart S, Pellier I, Armari-Alla C, Guitton C, Cheikh N, Kherfellah D, Vassal G, Thiebaut R, Laghouati S, Aladjidi N. Prospective Evaluation of the First Option, Second-Line Therapy in Childhood Chronic Immune Thrombocytopenia: Splenectomy or Immunomodulation. J Pediatr. 2021 Apr;231:223-230. doi: 10.1016/j.jpeds.2020.12.018. Epub 2020 Dec 17.

    PubMed 33340549

  • Pincez T, Fernandes H, Leblanc T, Michel G, Barlogis V, Bertrand Y, Neven B, Chahla WA, Pasquet M, Guitton C, Marie-Cardine A, Pellier I, Armari-Alla C, Benadiba J, Blouin P, Jeziorski E, Millot F, Paillard C, Thomas C, Cheikh N, Bayart S, Fouyssac F, Piguet C, Deparis M, Briandet C, Dore E, Picard C, Rieux-Laucat F, Landman-Parker J, Leverger G, Aladjidi N. Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden. Haematologica. 2022 Feb 1;107(2):457-466. doi: 10.3324/haematol.2020.271106.

    PubMed 33440924

  • Pincez T, Aladjidi N, Heritier S, Garnier N, Fahd M, Abou Chahla W, Fernandes H, Dichamp C, Ducassou S, Pasquet M, Bayart S, Moshous D, Cheikh N, Paillard C, Plantaz D, Jeziorski E, Thomas C, Guitton C, Deparis M, Marie Cardine A, Stephan JL, Pellier I, Dore E, Benadiba J, Pluchart C, Briandet C, Barlogis V, Leverger G, Leblanc T. Determinants of long-term outcomes of splenectomy in pediatric autoimmune cytopenias. Blood. 2022 Jul 21;140(3):253-261. doi: 10.1182/blood.2022015508.

    PubMed 35443028

  • Pincez T, Fernandes H, Pasquet M, Abou Chahla W, Granel J, Heritier S, Fahd M, Ducassou S, Thomas C, Garnier N, Barlogis V, Jeziorski E, Bayart S, Chastagner P, Cheikh N, Guitton C, Paillard C, Lejeune J, Millot F, Li-Thiao Te V, Mallebranche C, Pellier I, Neven B, Armari-Alla C, Carausu L, Piguet C, Benadiba J, Pluchart C, Stephan JL, Deparis M, Briandet C, Dore E, Marie-Cardine A, Leblanc T, Leverger G, Aladjidi N. Impact of age at diagnosis, sex, and immunopathological manifestations in 886 patients with pediatric chronic immune thrombocytopenia. Am J Hematol. 2023 Jun;98(6):857-868. doi: 10.1002/ajh.26900. Epub 2023 Mar 21.

    PubMed 36882195

  • Granel J, Fernandes H, Bader-Meunier B, Guth A, Richer O, Pillet P, Leverger G, Ducassou S, Fahd M, Pasquet M, Garnier N, Barlogis V, Guitton C, Jeziorski E, Thomas C, Bayart S, Cheikh N, Paillard C, Abou Chahla W, Chastagner P, Neven B, Millot F, Lejeune J, Li-Thiao Te V, Armari-Alla C, Briandet C, Carausu L, Deparis M, Piguet C, Benadiba J, Marie-Cardine A, Stephan JL, Pellier I, Pluchart C, Dore E, Michaux K, Heritier S, Leblanc T, Aladjidi N. Antinuclear antibody-associated autoimmune cytopenia in childhood is a risk factor for systemic lupus erythematosus. Blood. 2024 Apr 18;143(16):1576-1585. doi: 10.1182/blood.2023021884.

    PubMed 38227934

Questions about this trial

What is this clinical trial studying?

An observational study of Immune Thrombocytopenia and Autoimmune Hemolytic Anemia, run by University Hospital, Bordeaux at 9 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 10 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Nathalie ALADJIDI, MD (+33 557820261, nathalie.aladjidi@chu-bordeaux.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

AIC context (time frame: Baseline). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05937828, which also lists 12 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2023.

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